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Survey about achondroplasia
We have designed a questionnaire with the purpose to better know the population connected to achondroplasia.
The questionnaire is optional and anonymous. The data obtained will be used for statistical purposes and to better understand the natural history of achondroplasia. Please consider answering.
If you would like to take the survey later, you can click on the link at the top "Register at BA"

The soluble FGFR3, Therachon's TA- 46, may prevent early onset of obesity in achondroplasia

Developing during childhood, obesity is a common complication associated with achondroplasia, affecting more than 50% of people with this condition. Here we present a paper demonstrating that obesity in achondroplasia is markedly different from other kinds of obesity and is less prone to developing most of the complications associated with obesity. The authors have also demonstrated that treatment with soluble FGFR3 reduces weight and normalizes the metabolic disturbances associated with achondr […]

The International Society of Skeletal Dysplasia 2017 meeting report

We report on the 2017 International Skeletal Dysplasia Society (ISDS) meeting. Previously unknown metabolic disturbances were discovered in achondroplasia and new results were presented on meclazine for achondroplasia.

Recruitment in clinical trials

BioMarin shared a document with all associations updating everyone who is interested in their upcoming medications on their clinical trials. Read the whole document here.

Therachon heading to Phase 1 clinical trial

Therachon is developing a drug for achondroplasia, which has now crossed an important threshold in drug development for rare diseases. It has obtained Orphan Drug designation by the American and European regulatory agencies (FDA and EMA, respectively), taking it a step closer to a possible market authorization.

BioMarin updates the Multicenter and Multinational Clinical Study 111-901

This is an update on the observational study which collects information to establish a baseline for the other clinical trials by BioMarin. It is necessary to be enrolled in this one first for 6 months. Learn where to enroll here.

BioCentury published the article "Competing for growth"

Part of an article originally posted by Biocentury about the potential new therapies for achondroplasia and how the competition between the pharmaceutical companies is being beneficial for the final product.
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